On August 9, 2021 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2021 and provided a business update (Press release, Adaptimmune, AUG 9, 2021, View Source [SID1234586120]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With the data presented at ASCO (Free ASCO Whitepaper) for afami-cel and our planned BLA filing next year, I am pleased with progress on our ‘2-2-5-2’ strategy," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We will present data updates from our Phase 1 trials, SURPASS and ADP-A2AFP, at upcoming conferences and I am confident that Adaptimmune is well-placed to maintain its leadership position in cell therapies for cancer."

Upcoming confirmed data updates
ADP-A2AFP Phase 1 Trial at ILCA

On Sunday, September 5, 2021, Dr. Bruno Sangro of Clinica Universidad de Navarra will present data from Cohort 3 and the expansion phase of the Phase 1 trial of ADP-A2AFP in liver cancer during an oral presentation scheduled for 12:25 p.m. CET at the International Liver Cancer Association’s (ILCA) Annual Conference
As of the April 5th data cut-off used for ILCA, 13 patients had received ADP-A2AFP in Cohort 3 and expansion, 11 patients were evaluable with at least one post-baseline scan
SURPASS Phase 1 Trial at ESMO (Free ESMO Whitepaper)

The Company will present an update from its Phase 1 SURPASS trial in an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress that will be available online September 16th
As of the August 2nd data cut-off, 25 patients had received ADP-A2M4CD8, 23 patients had at least one post-baseline scan
The trial continues to recruit in lung, gastroesophageal, head and neck, and bladder cancers – focus indications, for which there have been early signs of efficacy, including responses, with SPEAR T-cells targeting MAGE-A4
Based on early data from patients with ovarian cancer treated in the trial, the Company is planning to add this as a focus indication to the SURPASS trial
SPEARHEAD-1 and afami-cel BLA

As presented at ASCO (Free ASCO Whitepaper) 2021, the Company reported an overall response rate for patients with at least one scan (evaluated by RECIST 1.1 per investigator assessment) of 39.3% (13/33), 41.4% (12/29) for synovial sarcoma, including two complete responses, and 25.0% (1/4) for MRCLS from its Phase 2 SPEARHEAD-1 trial
Of the 29 patients with synovial sarcoma, the disease control rate (defined as either response or stable disease) was 86.2% (25/29 patients) with 2 complete responses and 10 partial responses
This data set is intended to support planned BLA filing next year
Next data update planned at CTOS 2021
The European Medicines Agency and the FDA have agreed to Adaptimmune’s pediatric investigational plans
Working with key industry leaders to prepare for a successful commercial launch
Adaptimmune has partnered with Agilent for the development, manufacturing, and supply of a companion diagnostic for the MAGE-A4 biomarker
The Company is also working with Miltenyi Biotec for the process validation, manufacture, and supply of the lentiviral vector for use in the product for commercial launch
Further indications for next-gen SPEAR T-cell therapies
SURPASS-2

Planning to initiate a Phase 2 clinical trial with ADP-A2M4CD8, SURPASS-2, in esophageal and esophagogastric junction cancers in the third quarter of 2021
Other early-stage programs

The Company ceased enrollment in the Radiation Sub-Study of the afami-cel Phase 1 trial at the end of July. Five patients were treated in this sub-study and the Company plans to provide a data update at an upcoming congress
Corporate

The Company received a development milestone payment of $4.2 million in the three months ended June 30, 2021
Financial Results for the three and six months ended June 30, 2021

Cash / liquidity position: As of June 30, 2021, Adaptimmune had cash and cash equivalents of $50.5 million and Total Liquidity1 of $285.4 million.
Revenue: Revenue for the three and six months ended June 30, 2021 was $3.1 million and $3.5 million, respectively, compared to $0.5 million and $1.3 million for the same periods in 2020. Revenue has increased primarily due to an increase in development activities under the Astellas Collaboration Agreement.
Research and development (R&D) expenses: R&D expenses for the three and six months ended June 30, 2021 were $28.9 million and $53.4 million, respectively, compared to $20.5 million and $41.7 million for the same periods in 2020. R&D expenses increased due to an increase in the number of employees engaged in research and development, and increases in costs related to the development of a companion diagnostic assay and our Phase 2 clinical trial associated with ADP-A2M4CD8. These increases were partially offset by an increase in reimbursements receivable for research and development tax and expenditure credits.
General and administrative (G&A) expenses: G&A expenses for the three and six months ended June 30, 2021 were $13.5 million and $27.4 million, respectively, compared to $10.3 million and $19.6 million for the same periods in 2020 due to increases in employee-related costs, share-based compensation expense, and professional fees.
Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three and six months ended June 30, 2021 was $39.1 million and $76.8 million respectively ($(0.04) and $(0.08) per ordinary share), compared to $29.9 million and $58.0 million ($(0.04) and $(0.07) per ordinary share) for the same periods in 2020.
Financial Guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into early 2023, as further detailed in the Company’s Quarterly Report on Form 10-Q for the three and six months ended June 30, 2021, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information
The Company will host a live teleconference and webcast to provide additional details at 4:30 p.m. EDT (9:30 p.m. BST) today. A live webcast of the conference call and replay can be accessed at https://bit.ly/3zFas59. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (833) 652-5917 (US or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (7867634).

On August 9, 2021 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported the receipt of notification from its partner, AbbVie Inc., regarding its termination of the license and collaboration agreement for the investigational drug abicipar pegol for the treatment of neovascular age-related macular degeneration (nAMD) and Diabetic Macular Edema (DME) (Press release, Molecular Partners, AUG 9, 2021, View Source [SID1234586136]). As such, Molecular Partners will regain the development and commercial rights of abicipar on a worldwide basis.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"There remains a significant unmet medical need for patients living with nAMD and DME, and we remain confident in abicipar’s potential to offer these patients a differentiated treatment option over existing therapies," said Patrick Amstutz, Chief Executive Officer of Molecular Partners. "Our focus for this program will be determining the best path to value creation within the context of our expansive portfolio of antiviral and immuno-oncology therapies in development."

Molecular Partners will form a special committee to evaluate the program and determine appropriate next steps. In addition, Molecular Partners and AbbVie will continue their ongoing discovery alliance, in which AbbVie will continue to evaluate additional DARPin candidates for ophthalmic indications. The return of the abicipar program is not expected to impact Molecular Partners’ financial outlook for 2021 or previously issued guidance.

Abicipar is a long-acting anti-VEGF DARPin molecule which was invented by Molecular Partners and initially licensed to Allergan in 2011. The program has been through two positive Phase 3 studies, CEDAR and SEQUOIA, which supported the non-inferior efficacy of the abicipar quarterly dosing regimen to maintain vision gains with more than 50 percent fewer injections versus ranibizumab (13 vs. 6) dosed monthly in the first year.

With the acquisition of Allergan by AbbVie, the rights to abicipar were transferred to AbbVie. In June 2020, AbbVie received a Complete Response Letter to the Biologics License Application for abicipar pegol, indicating that the rate of intraocular inflammation observed following administration of Abicipar pegol resulted in an unfavorable benefit-risk ratio in the treatment of nAMD (AMD), and that additional work would be required to demonstrate a lower rate of ocular inflammation than what was previously seen in the Phase 3 studies.

On August 9, 2021 Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer, reported guidance on the development timeline for its lead product candidate, momelotinib (Press release, Sierra Oncology, AUG 9, 2021, View Source [SID1234586153]). The company now expects topline data from the Phase 3 registration-enabling MOMENTUM study by February 2022, and assuming positive results, the company plans to file a New Drug Application with the U.S. Food & Drug Administration in Q2 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sierra also announced it has raised $34 million in gross proceeds thus far in Q3 2021 through the use of its at-the-market (ATM) offering. These funds are in addition to the $90.7 million in cash and cash equivalents on hand at the close of the second quarter of 2021, further bolstering the company’s cash balance as it prepares for momelotinib commercialization. The company also has the potential to obtain up to $33.3 million in cash from Series B warrants that must be exercised within 75 days of the announcement of MOMENTUM topline data.

Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology noted, "The continued excellent execution of the MOMENTUM study and further acceleration of the timeline, combined with the additional funding secured over the last few weeks, puts us in a very strong position as we approach topline data."

On August 9, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the second quarter ended June 30, 2021. In addition, the Company provided a clinical and business update (Press release, Syndax, AUG 9, 2021, View Source [SID1234586105]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The second quarter of 2021 was marked by significant progress advancing our pipeline of innovative therapeutics, and we are pleased to share that we have selected a go-forward dose for the pivotal Phase 2 portion of AUGMENT-101 based on favorable findings from the intermediate dose cohort," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Enrollment in the trial continues, with recently enrolled patients expected to count towards the Phase 2 expansion portion, subject to endorsement of the Phase 2 dose by the U.S. Food and Drug Administration (FDA). We are also excited to announce an important step in our plans to expand development of SNDX-5613 into earlier lines of therapy with the initiation of the first frontline combination trial of a menin inhibitor in the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial."

"Beyond SNDX-5613, enrollment remains on track in the ongoing global pivotal AGAVE-201 trial of axatilimab in patients with chronic graft versus host disease (cGVHD), and we continue to expect topline data from the trial in 2023. In addition, as previously announced, we anticipate sharing updated results from the recently completed Phase 1/2 trial of axatilimab in cGVHD later this year and remain committed to unlocking the full potential of axatilimab in the growing number of cGVHD patients lacking effective interventions."

Recent Progress and Anticipated Milestones

SNDX-5613

In May 2021, the Company reported updated data from the ongoing Phase 1 dose escalation portion of the Phase 1/2 AUGMENT-101 trial of SNDX-5613, a highly selective oral menin inhibitor, in patients with mixed lineage leukemia rearranged (MLLr) and nucleophosmin (NPM1c) mutant relapsed/refractory (R/R) acute leukemias. The updated May data showed that a total of 7/31 patients (23%) have achieved CR/CRh.

Based on positive findings from the intermediate dose cohort, in which no dose limiting toxicities were observed, the Company has selected 276 mg of SNDX-5613 every 12 hours in patients who are not receiving a concomitant strong CYP3A4 inhibitor (Arm A) and 163 mg every 12 hours for patients who are receiving a concomitant strong CYP3A4 inhibitor (Arm B), for Phase 2. Enrollment in the trial remains ongoing, with recently enrolled patients expected to count towards the Phase 2 expansion portion, subject to endorsement of the Phase 2 dose by the U.S. FDA.

The Company reported it will initiate a frontline trial of SNDX-5613 in combination with venetoclax and azacytidine in newly diagnosed acute myeloid leukemia (AML) patients unable to tolerate induction chemotherapy. The trial will be conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial, a collaborative clinical trial that aims to change the paradigm of AML treatment through a precision medicine approach. SNDX-5613 is the first menin inhibitor to be included in the Beat AML Master Clinical Trial.

The Company also announced today that it plans to initiate a new trial to assess the safety, tolerability, and preliminary anti-leukemic efficacy of SNDX-5613 in combination with chemotherapy in patients with R/R MLLr or NPM1 acute leukemias. The Phase 1b trial, which will be referred to as AUGMENT-102, is expected to enroll up to 27 patients.

In June 2021, the Company announced that the U.S. FDA granted Fast Track Designation (FTD) to SNDX-5613 for the treatment of adult and pediatric patients with R/R acute leukemias harboring an MLLr or NPM1 mutation. FTD is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier.
Axatilimab

In May 2021, Syndax announced that enrollment is complete in the Phase 2 expansion portion of the Phase 1/2 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with cGVHD. The Company continues to anticipate reporting updated results at a medical meeting in the fourth quarter of 2021 for 40 patients, including the 17 patients in the Phase 1 portion and 23 patients from the Phase 2 expansion portion, which evaluated 1 mg/kg of axatilimab every two weeks. At the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020, preliminary data from the Phase 1 portion of the trial were reported during an oral presentation highlighting the tolerability and high response rate of axatilimab in cGVHD patients refractory to multiple therapeutic agents.

Enrollment is ongoing in the Company’s global pivotal Phase 2 AGAVE-201 trial of axatilimab in patients with cGVHD, with topline data expected in 2023. The trial will evaluate the safety and efficacy of three doses and schedules of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score.

Earlier this year, the Company announced that the U.S. FDA granted Orphan Drug Designation to axatilimab for the treatment of patients with cGVHD and idiopathic pulmonary fibrosis.
Second Quarter 2021 Financial Results

As of June 30, 2021, Syndax had cash, cash equivalents and short-term investments of $253.1 million and 51.9 million shares and share equivalents issued and outstanding. This includes 3.3 million pre-funded warrants.

Second quarter 2021 research and development expenses increased to $16.9 million from $10.9 million for the prior year period. The increase was primarily due to increased clinical trial activities and increased CMC activities.

General and administrative expenses for the second quarter 2021 decreased to $5.8 million from $6.0 million for the prior year period. The decrease is primarily due to the absence of pre-commercialization expenses for entinostat in 2021 partially offset by employee related expenses.

For the three months ended June 30, 2021, Syndax reported a net loss attributable to common stockholders of $22.9 million or $0.44 per share compared to $17.1 million or $0.42 per share for the prior year period.

Financial Update and Guidance

For the third quarter of 2021, research and development expenses are expected to be $25 to $30 million, and total operating expenses are expected to be $30 to $35 million. For the full year of 2021, research and development expenses are expected to be $90 to $100 million, and total operating expenses are expected to be $110 to $120 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, August 9, 2021.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following

On August 9, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported its financial results for the second quarter ended June 30, 2021 and provided an update on recent developments (Press release, IGM Biosciences, AUG 9, 2021, View Source [SID1234586121]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to make steady progress in the development of our innovative product pipeline, including the successful completion of the initial dose escalation portion of our IGM-2323 Phase 1 clinical trial," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We look forward to announcing a recommended Phase 2 dose for IGM-2323, presenting initial dose escalation data from the Phase 1 trial of IGM-8444, initiating a Phase 1 trial for IGM-6268 and filing an IND for IGM-7354 this year. "

Pipeline Updates

IGM-2323

Phase 1 dose escalation completed; dose expansion continues. IGM continues to advance the clinical development of IGM-2323, the Company’s IgM-based CD20 x CD3 bispecific antibody, for the treatment of non-Hodgkin’s lymphoma (NHL). The Company cleared its highest planned dose escalation cohort, a top dose of 1000 mg, without a dose limiting toxicity, and is currently treating additional patients in four Phase 1 dose cohorts with top doses of 100 mg, 300 mg, 600 mg and 1000 mg, respectively. IGM expects to select a recommended Phase 2 dose in 2021.
IGM-8444

Additional dose cohorts cleared in Phase 1. IGM continues to advance the clinical development of IGM-8444, the Company’s IgM Death Receptor 5 (DR5) agonist, for the treatment of a potentially broad range of solid tumors and hematologic malignancies. IGM has cleared its third dose cohort (3 mg/kg) of the single-agent portion of its Phase 1 clinical study and is currently treating patients in its highest dose escalation cohort (10 mg/kg) with every two-week single agent dosing. IGM has also cleared the first dose cohort of the FOLFIRI combination portion of the Phase 1 study and is currently treating patients in the second of four planned FOLFIRI combination dose escalation cohorts. IGM expects to report initial data from the dose escalation portion of the Phase 1 trial in 2021.
Clinical testing of birinapant in combination with IGM-8444 expected to begin this year. IGM remains on track to begin clinical testing of birinapant in combination with IGM-8444 in 2021.
IGM-6268

New pipeline candidate for the treatment and prevention of COVID-19 expected to advance into the clinic this year. In June 2021, IGM announced IGM-6268, which represents the expansion of the Company’s IgM platform into infectious diseases. IGM-6268 is an IgM version of an anti-SARS-CoV-2 IgG monoclonal antibody and is being developed as an intranasally administered agent for the treatment and prevention of COVID-19. IGM expects to initiate a Phase 1 clinical trial of IGM-6268 in 2021.
Nature manuscript published. In June 2021, Nature published an article entitled "Nasal delivery of an IgM offers broad protection from SARS-CoV-2 variants". The article describes results from preclinical studies demonstrating significantly greater neutralization of SARS-CoV-2 with an IgM antibody compared to IgG antibodies, the potent neutralization of all evaluated mutant Variants of Concern and Variants of Interest, and the ability to provide effective preventative and therapeutic protection when delivered intranasally in mice. The article was co-authored by researchers at IGM, The University of Texas Medical Branch at Galveston and The University of Texas Health Science Center at Houston.
IGM-7354

Investigational New Drug (IND) application expected to be filed this year. IGM plans to file an IND application with the U.S. Food and Drug Administration (FDA) for IGM-7354, the Company’s IL‑15 x PD‑L1 bispecific IgM antibody, before the end of 2021 in order to begin clinical testing initially in solid tumors, followed by hematologic malignancies.
Corporate Updates

Chris Takimoto appointed Chief Medical Officer. Dr. Takimoto brings 30 years of experience in cancer research and development. Most recently, Dr. Takimoto was Senior Vice President, Oncology at Gilead Sciences, Inc. Prior to Gilead, he served as Chief Medical Officer of Forty Seven, Inc., a biotechnology company formed out of Stanford University and acquired by Gilead Sciences in 2020.
Second Quarter 2021 Financial Results

Cash and Investments: Cash and investments as of June 30, 2021 were $301.8 million, compared to $366.3 million as of December 31, 2020.
Research and Development (R&D) Expenses: For the second quarter of 2021, R&D expenses were $30.1 million, compared to $15.0 million for the same period in 2020.
General and Administrative (G&A) Expenses: For the second quarter of 2021, G&A expenses were $8.6 million, compared to $4.4 million for the same period in 2020.
Net Loss: For the second quarter of 2021, net loss was $38.7 million, or a loss of $1.16 per share, compared to a net loss of $18.8 million, or a loss of $0.62 per share, for the same period in 2020.
2021 Financial Guidance

IGM reiterates its previously issued financial guidance expecting full year GAAP operating expenses to be between $175 million and $185 million including estimated non-cash stock-based compensation expense of approximately $25 million. IGM expects to end 2021 with a balance of over $200 million in cash and investments.